BENEFITING: AUBURN UNIVERSITY FOUNDATION
EVENT DATE: Feb 23, 2015
The Auburn University College of Veterinary Medicine, in cooperation with the Auburn University Foundation, is using a crowdsourcing site to raise awareness and financial support for its GM1 research.
Associate Professor Dr. Doug Martin said he believes that if funding is raised through the site, CrowdRise, there could be a breakthrough in treatment for the rare, fatal neurological disease.
“If we could get started on the studies that could lead to eventual FDA approval, we believe we could see real results in clinical trials,” he said.
Translating Animal Medicine Breakthroughs
into Human Medicine Cures
For decades, the Scott-Ritchey Research Center in Auburn’s College of Veterinary Medicine has researched GM1 gangliosidosis in felines, as GM1 occurs naturally in cats. The goal is to find a cure for feline GM1 and, through research partners across the United States, successfully apply similar therapies that translate into human medicine.
Martin and his team at Scott-Ritchey have successfully extended the life expectancy of cats by more than five times compared to non-treated GM1 cats with the use of gene therapy, a non-harmful viral vector to produce enzymes missing in GM1 and Tay-Sachs-related diseases.
Treated cats are living years past the life expectancy of non-treated cats and their survival time continues to increase. Martin’s goal is to model that same success in human clinical trials.
“We believe 100 percent that the research we are doing will help children like Porter Heatherly,” said Martin.
Porter Heatherly, the son of Auburn alumni Sara and Michael Heatherly, has GM1 gangliosidosis, an inherited disease that progressively destroys nerve cells in the brain and spinal cord. There is no known cure and unfortunately for children like Porter, who have infantile-onset GM1, the life expectancy is just two years. Porter turned 2 on Sept. 14. The Heatherlys hope Porter’s story will inspire support for Martin’s research.
An International Partnership
Auburn is part of an international team – the Tay-Sachs Gene Therapy Consortium – established in 2007 by researchers from Scott-Ritchey, Cambridge University, the University of Massachusetts Medical School, Boston College, Massachusetts General Hospital and New York University. The consortium has pooled its efforts to devise the most effective gene therapy approach to treat Tay-Sachs disease and bring it into simultaneous clinical trials in the U.S. and U.K.
Martin believes the research is ready for clinical trials in humans, but before that can happen, the college must secure $500,000 in funding so Scott-Ritchey scientists can pursue research leading to studies needed for FDA approval.
Supporting Auburn's GM1 Research
Donations in support of Auburn's GM1 research being performed in the Scott-Ritchey Research Center may be made:
- online through CrowdRise at www.crowdrise.com/offerporterhopecureg/fundraiser/auburnuniversityfoun or
- online directly to the Auburn University Foundation at https://cws.auburn.edu/giving/Default.aspx?allocation=30090924
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